Detalhe da pesquisa
1.
Current Clinical Applications of In Vivo Gene Therapy with AAVs.
Mol Ther
; 29(2): 464-488, 2021 02 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-33309881
2.
Gauging Gait Disorders with a Method Inspired by Motor Control Theories: A Pilot Study in Friedreich's Ataxia.
Sensors (Basel)
; 21(4)2021 Feb 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-33562027
3.
Longitudinal changes of swallowing safety and efficiency in infants with spinal muscular atrophy who received disease modifying therapies.
Pediatr Pulmonol
; 59(5): 1364-1371, 2024 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-38358081
4.
Thrombotic microangiopathy following systemic AAV administration is dependent on anti-capsid antibodies.
J Clin Invest
; 134(1)2024 Jan 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-37988172
5.
Polysomnography findings in children with spinal muscular atrophy after onasemnogene-abeparvovec.
Sleep Med
; 101: 234-237, 2023 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-36442421